Patients and Families

A clear purpose

The diseases for which we develop therapies provide inspiration for our day-to-day activities. There is clarity in purpose, a collective empathy driving us forward. Krystal was named after this clarity — a simple realization that we can do better. In doing so, we extend gratitude to the patients, caregivers, advocates and physicians dedicated to the diseases which unite us in purpose.

The health and well-being of our patients is our highest priority, and bringing innovative, effective, and non-invasive treatment options to them is our ultimate goal. We strive to accomplish this by communicating extensively with patients, their caregivers, and families to better understand the hardships, wants, and needs of those battling debilitating skin diseases. In addition, we interact regularly with a number of patient advocacy groups, including the Dystrophic Epidermolysis Research Association (Debra) of America, the Epidermolysis Bullosa Medical Research Foundation (EBMRF), and the Epidermolysis Bullosa Research Partnership (EBRP), to support patient rights, awareness, and education.

How our gene therapy works

The gene therapies that Krystal Biotech is developing are designed to introduce therapeutic genes into a patient's cells. The genes are introduced into these cells using a modified HSV-1 virus that has been engineered to render the virus harmless to the patient. Specifically, the virus is modified so that it will not replicate in the patient's cells. In addition, the new genes that are introduced by the virus do not integrate into the cell's native genetic material. Once the modified viral vector has delivered the genes into the skin cells, the genes then make (express) the encoded functional protein, supplementing the non-functional proteins expressed from the patient's own genome, thereby providing treatment to the underlying source of the disease. We call this our "STAR-D" technology and we are using it initially to develop topical treatments for rare or orphan dermatological indications. Learn more about the STAR-D platform here.